Autologous stem cell transplants in treatment of multiple sclerosis: where we stand and future prospects

Based on experimental and clinical observations, high-dose immunosuppression followed by autologous transplantation may induce remissions in severe, refractory, autoimmune disorders including multiple sclerosis, a disease which, in its progressive form, does not respond to treatment. Phase I/II studies of transplantation in MS published by individual centers as well as a comprehensive analysis of the reports to the EBMT registry have shown that transplantation may positively affect MS by stabilizing the clinical condition of the patients, by improving their disability status, and by completely abrogating the inflammatory process in the brain as evidenced in magnetic resonance imaging. Other available therapies do not appear to be so efficacious as transplantation. However, the procedure is associated with a transplant-related mortality risk of about 3 to 8%. Therefore, it cannot be recommended for the treatment of a chronic, non-lethal, disease like MS unless it proves superior to standard therapies in terms of efficacy. This can be demonstrated only in a randomized trial, which is being launched by the EBMT under the name ASTIMS. It compares the BEAM regimen plus autotransplantation to mitoxantrone, which is currently regarded as one of the best available treatments, in patients with secondary progressive or rapidly evolving relapsing/remitting multiple sclerosis.